Section 3 of 6
Product information and disease description
73 evidence topics · 66 sources
Product description
Phase of product development
Fast Track and Breakthrough Therapy designations
Regulatory submission: BLA resubmitted December 2025
Launch
No evidence found.
Approval status in the second-quarter business update
European regulatory route in the horizon scan
Product information
Generic, brand name and therapeutic class of product
Proposed trade name and product name
Dosage forms and strengths
IMPALA-2 investigational formulation
| Attribute | Reported value |
|---|---|
| Dosage form | “Nebulizer solution” |
| Concentration | “250 µg/mL” |
Material safety data sheet
No evidence found.
Average sales price and wholesale acquisition cost
Not applicable.
American hospital formulary service (AHFS), or other drug classification
ATC classification
| Attribute | Reported value |
|---|---|
| Code | “L03AA03” |
| Substance | “molgramostim” |
| Class | “Colony stimulating factors” |
Indication
Pharmacology
Mechanism of action
GM-CSF regulation of surfactant homeostasis and host defense
Pharmacodynamics
Healthy volunteers: blood leukocyte response
Pharmacokinetics
Healthy volunteers: systemic exposure and elimination
Healthy volunteers: exposure proportionality
Contraindications/Warnings/Precautions/Adverse effects
Warnings and precautions
Not applicable.
Special populations
Pregnancy and breastfeeding: IMPALA-2 exclusions
Renal impairment
No evidence found.
Hepatic impairment
No evidence found.
Drug/Drug, drug/disease interactions
Effects of other drugs on molgramostim
No evidence found.
Effects of molgramostim on other drugs
No evidence found.
Dosing and administration
Dosage
IMPALA-2 investigational dose
Administration
Access and distribution
Anticipated specialty pharmacy distribution and patient assistance
Co-prescribed/Concomitant therapies
Effect of molgramostim on quality measures
No evidence found.
Product comparison
Sargramostim and molgramostim: limits of indirect comparisons
Daratumumab in refractory disease: case report
Place of product in therapy
Disease description
Definition and etiology
Surfactant accumulation in autoimmune pulmonary alveolar proteinosis
Epidemiology
Incidence of Autoimmune pulmonary alveolar proteinosis
Japanese registry: minimum incidence and prevalence
Prevalence of Autoimmune pulmonary alveolar proteinosis
United States claims analysis: all PAP subtypes
Updated United States claims-based autoimmune PAP population estimate
Natural history, survival, and mortality
Pathophysiology
Diagnosis
Serum GM-CSF neutralizing autoantibodies
Immunochromatographic anti-GM-CSF autoantibody testing
Exploratory biomarker study population
Clinical presentation - signs and symptoms
Respiratory symptoms: dyspnea, cough, and sputum production
Functional symptoms: fatigue and reduced exercise capacity
Long-term morbidity
Progression to pulmonary fibrosis
Burden of Autoimmune pulmonary alveolar proteinosis
Humanistic burden and health-related quality of life
Reported burden of current treatments and off-label therapies
Economic burden and healthcare resource utilization
United States claims analysis: PAP and matched-control cohorts
| Measure | PAP cohort | Control cohort |
|---|---|---|
| Participants | “2312” | “9247” |
| Average charges during follow-up, mean ± standard deviation | “$71,672.64 ± $226,117.54” | “$14,655.91 ± $74,791.03” |
Hospital admissions: nationwide inpatient sample
Economic impact of Autoimmune pulmonary alveolar proteinosis on families
No evidence found.
Economic impact of diagnostic testing
Serum GM-CSF autoantibody testing: qualitative cost rationale
Approaches to treatment
Current treatment options and standard of care
Whole lung lavage
Historical standard of care
Restoration of alveolar function without cure
Inhaled Granulocyte-Macrophage Colony-Stimulating factor
Nebulized GM-CSF systematic review: serious adverse events
Mild-to-moderate disease trial: primary endpoint
Long-term inhaled GM-CSF: relapse during dose de-escalation
B-cell depletion with rituximab
Plasmapheresis
Lung transplantation
Supportive care and exposure reduction
Limitations of current therapies
Summary
Whole lung lavage can restore alveolar function but does not cure the disease. The ERS recommendation for inhaled GM-CSF is strong, but its stated evidence certainty is very low. Relapse during inhaled GM-CSF dose de-escalation has been reported. Indirect comparisons of sargramostim and molgramostim are exploratory and do not establish clinical equivalence.
Place in treatment, anticipated use, and care setting
Summary
Guideline-based first-line options for symptomatic or progressive autoimmune pulmonary alveolar proteinosis are whole lung lavage and inhaled GM-CSF. The studied molgramostim regimen is 300 µg daily using a dedicated investigational eFlow nebulizer system. Expanded access requests must come from a licensed pulmonologist and do not guarantee treatment access. Savara has selected an exclusive U.S. specialty pharmacy, with commercial distribution and patient assistance contingent on approval.
Heterogeneity of treatment effect
IMPALA-2 treatment effect across baseline DLCO subgroups
Care management intervention strategies
Referral for diagnostic and therapeutic guidance
Other product development or post-marketing obligations required by the FDA
Not applicable.
Ongoing post-approval monitoring
Not applicable.